FDA gives approval to new leukemia drug

A new drug that helped more than 90 percent of patients with a rare form of leukemia won government approval in record time Thursday and was hailed as "the wave of the future" in fighting cancer.

"This new drug, we believe, is a picture of the future of cancer treatment," said Dr. Richard D. Klausner, director of the National Cancer Institute.

Food and Drug Administration approval of Gleevec was announced by Health and Human Services Secretary Tommy G. Thompson at a news conference, an unusual step for a drug approval.

Gleevec, made by Novartis Pharmaceuticals, is specifically targeted at chronic myeloid leukemia, a disease that claims about 2,300 American lives annually.

For the past four months, Leon Watson of Fritch has popped the powder capsules. In October, Watson injured his leg, and after surgery, doctors told him he had leukemia.

Dr. Phillip Periman, Watson's doctor from the Don and Sybil Harrington Cancer Center, suggested Watson travel to the MD Anderson Cancer Center in Houston to try out for the clinical study.

"I just had this calmness about it," Watson said. "When I went down I felt pretty confident that I would be accepted, and of course, there was a sigh of relief."

Since December, Watson takes four pills in the morning with his breakfast. He travels to Houston once a month to meet with doctors. Watson keeps daily reports of how he feels so physicians can detail any side effects. So far, it seems like aspirin, he said.

"I think this is just a huge breakthrough," Watson said. "I hope it is the beginning of the end for more than just leukemia, but to give everybody hope."

Watson said he will continue taking the pills for a year.

"This is a major deal," Periman said. "I'm thankful they approved it, and fortunately over the last 10 years, the FDA has learned to be more quick with cancer and AIDS drugs because if you wait the usual time, the patient is dead."

The drug has been about 97 percent effective, but the long-term effects are not known, Periman said.

Thompson said the drug is based on the principle of molecular targeting, killing leukemia cells while leaving normal white cells alone.

"We believe such targeting is the wave of the future," Thompson said.

Dawn Willis, scientific programs director for the American Cancer Society, also praised the approval.

"I can't think of another cancer drug that has shown such spectacular success in a group of people that had failed all other treatment," she said. She said the concept of attacking cancer by designing a small molecule that will specifically attack a target protein should be usable in other forms of the disease as well.

Gleevec itself is being tested against about a dozen forms of the disease and researchers are working on other similar targeting drugs.

Dr. Daniel Vasella, president of Novartis AG, the Swiss parent company, said he hopes to begin shipping the drug by next Monday and to have it in pharmacies within a week.

U.S. shares of Novartis were up $3.09, or 8 percent, to $42.09 in afternoon trading on the New York Stock Exchange.

The one-pill-a-day regimen is expected to cost between $2,000 and $2,400 monthly, which company officials said is comparable to other current cancer treatments.

But Vasella said the company is setting up a program to make sure it is available to uninsured, low-income people. He said it would be priced at a sliding scale for people with incomes below $100,000 a year and would be free for those under $40,000 income.

Gleevec works by blocking chemical signals sent by cancerous cells and researchers hope it will also prove useful in a form of stomach tumor and perhaps other types of cancer as well.

The results of clinical trials of the drug, also known as STI-571 or imatinib mesylate, generated excitement among cancer researchers.

Chronic myeloid leukemia is caused by a protein that produces an abnormal chromosome. It leads to a huge increase in the number of white blood cells in the body, which can interfere with the functioning of other organs.

In clinical trials financed by Novartis, more than 90 percent of patients in the first phase of CML saw their cancer go into remission within the first six months of taking the pill, according to findings presented in December at a meeting of the American Society of Hematology.

A study of patients in the second phase of the disease showed more than 90 percent of those patients responded positively to the treatment, and in 63 percent, the cancer went into remission. The trials involved 530 first-phase and 230 second-phase patients.

The early success has propelled researchers to test the drug on almost 3,000 patients around the world.

Gleevec blocks a signal that the abnormal protein sends out, preventing the abnormal growth and production of other cancerous cells. It targets three specific signaling chemicals, some of which also are present in other forms of cancer.

Currently the only treatments for CML are bone marrow transplants, which can be dangerous, and interferon, which can extend a leukemia patient's life by up to two years but can have side effects that cause about 20 percent of patients to stop using it.

Gleevec has only been studied on humans for about two years, so how long it will prolong a patient's life is not yet known. But it has had few side effects.

Dr. Bernard A. Schwetz of the FDA termed Gleevec "an unprecedented drug with a new form of action," but stressed that long-term effects remain unknown.

Much remains to be learned as more people use the drug, agreed Klausner, but he said Gleevec "is as interesting and impressive as any (drug) we have seen in our long war on cancer."

Suzanne Dreger of Falls Church, Va., testified to its value.

She was diagnosed with CML four years ago and by last year interferon was no longer helping her, she said. Many days she was unable to even get out of bed until she was able to get into a trial of Gleevec, she said.

"It's been a great year for me," she beamed, reporting she was back at work and her cancer is near remission.